Motor neurone disease (MND): investigational drug treatments

Evidence-based neurology checklist on motor neurone disease (mnd): investigational drug treatments: Repurposed treatments Anakinra Biotin (MD1003) Fingolimod Ibudilast Melatonin Rasagiline Stem cell therapy Tamoxifen Other investigational treatments Failed investigational treatments

Repurposed treatments

  • Anakinra
  • Biotin (MD1003)
  • Fingolimod
  • Ibudilast
  • Melatonin
  • Rasagiline
  • Stem cell therapy
  • Tamoxifen

Other investigational treatments

Failed investigational treatments

References

  1. Khalid SI, Ampie L, Kelly R, Ladha SS, Dardis C. Immune modulation in the treatment of amyotrophic lateral sclerosis: a review of clinical trials. Front Neurol 2017; 8:486.
  2. Juntas-Morales R, Pageot N, Bendarraz A, et al. High-dose pharmaceutical grade biotin (MD1003) in amyotrophic lateral sclerosis: a pilot study. EClinicalMedicine 2020; 19:100254.
  3. Berry JD, Paganoni S, Atassi N, et al. Phase IIa trial of fingolimod for amyotrophic lateral sclerosis demonstrates acceptable acute safety and tolerability. Muscle Nerve 2017; 56:1077-1084.
  4. Bald EM, Nance CS, Schultz JL. Melatonin may slow disease progression in amyotrophic lateral sclerosis: findings from the Pooled Resource Open-Access ALS Clinic Trials database. Muscle Nerve 2021 (Online ahead of print).
  5. Ludolph AC, Schuster J, Dorst J, et al; RAS-ALS Study Group. Safety and efficacy of rasagiline as an add-on therapy to riluzole in patients with amyotrophic lateral sclerosis: a randomised, double-blind, parallel-group, placebo-controlled, phase 2 trial. Lancet Neurol 2018; 17:681-688.
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